GAINESVILLE, Fla.--(BUSINESS WIRE)-- Applied Genetic Technologies Corporation, (AGTC), a privately-held, clinical stage biotechnology company developing gene therapy products to treat rare retinal diseases, announced today that it has secured $37.5 million in a Series B round of financing.

Alta Partners and S.R. One, Limited led the financing, with new investor Osage University Partners joining existing investors InterWest, Intersouth Partners and MedImmune Ventures in the round. The funding will allow AGTC to continue development of its Phase 2 program in Alpha-1 Antitrypsin Deficiency (Alpha-1) and initiate full development of potential treatments for two orphan ophthalmology indications, Achromatopsia (ACHM) and X-Linked Rentinoschisis (XLRS).

"We are strong believers in the business model of developing treatments for genetic disorders," said Ed Hurwitz, General Partner of Alta Partners. "Based on encouraging clinical results from AGTC and others, we concluded that a large set of genetically defined diseases could be cured using AGTC’s proprietary vectors and manufacturing technologies. The Series B financing is designed to move several of AGTC’s programs through proof of concept as well as to allow the company to leverage its manufacturing and development infrastructure with partners to accelerate a broad portfolio of curative products.”

ACHM is an inherited genetic condition that presents at birth with impaired visual acuity. Most patients are legally blind, lacking color discrimination and experiencing extreme light sensitivity, resulting in daytime blindness. ACHM is caused by mutations in a group of genes which make the cone cells concentrated in the central retina non-functional. There is no treatment for Achromatopsia, although deep red tinted spectacles or contact lenses can reduce symptoms of light sensitivity. Approximately 22,000 patients in the US and Europe suffer from this disease.

AGTC’s potential treatment uses an adeno-associated virus (AAV), a safe, man-made virus that delivers healthy copies of the ACHM gene to the cells of the retina, replacing the defective copies of the gene. A single treatment is expected to halt the disease for several years, perhaps a lifetime. The AAV delivery system is successfully being used in clinical trials of Leber congenital amaurosis gene therapy that have restored vision in more than 50 adults and children who were virtually blind. Previous research has shown promising signs of efficacy in dog models of ACHM.

XLRS, an inherited genetic condition, is a leading cause of juvenile macular degeneration in males. It is caused by mutations in the RS1 gene, which results in the layers of the central retina splitting. Patients typically begin to experience progressive vision loss between the ages of 5 and 10. Other early symptoms include the inability to focus both eyes and roving, involuntary eye movements. No treatment for XLRS is currently available. Approximately 35,000 patients in the US and Europe suffer from this disease. Previous research has shown promising signs of efficacy in rodent models of XLRS.

About AGTC

AGTC is focused on the research and development of novel therapeutics for patients with unmet medical needs utilizing AGTC’s proprietary, non-pathogenic adeno-associated virus (AAV) delivery system. AGTC has demonstrated that this system can be used to deliver a normal form of a gene in both animals and humans thus allowing their own body to produce sustained therapeutic levels of important biologics. The Company’s most advanced programs in development are treatments for Alpha-1 antitrypsin deficiency (Alpha-1) a disease causing a progressive loss of lung function, and Leber’s Congenital Amaurosis, an inherited condition causing early blindness. Both utilize AGTC’s proprietary AAV system and production methods.

About Alta Partners

Alta Partners is a San Francisco-based venture capital firm focused on life sciences investing. Founded in 1996, the firm manages $2 billion in committed capital through eight venture fund programs. Alta invests in life sciences companies across the development continuum, from company formation to later-stage opportunities, and has funded more than 145 companies in the sector to date.

About S.R. One, Limited

S.R. One is the independent corporate venture capital arm of GlaxoSmithKline. The firm invests globally in emerging life science companies that are pursuing innovative science which will significantly impact medical care. Since 1985 S.R. One has invested in more than 150 companies, and its current portfolio includes approximately 30 private and public companies.

About Osage University Partners

Osage University Partners is a venture capital fund that invests exclusively in spinouts derived from a network of 46 affiliated research institutions. OUP partners with high quality universities to serve as their co-investment vehicle in start-ups that are licensing their technology. OUP invests across all sectors of university commercialization, including life sciences, physical sciences, and information technology. Osage invests broadly across stage, from seed to late stage, in venture capital rounds of start-ups alongside new lead investors.